Rare diseases affect an estimated 300 million people worldwide and present unique clinical development challenges. Advances in therapeutic science, trial design, and regulatory frameworks are reshaping the rare disease landscape, offering hope to patients and families with limited treatment options.
In this Q&A series, we spotlight the experts behind our rare disease clinical trials, exploring their career journeys and perspectives on the evolving field. In this edition, we sat down with Catalina Olano-Marín, Clinical Project Director, Clinical Trial Management, to discuss her experience and the role that Medpace plays in advancing innovative rare disease therapies.
Tell us about your background. What motivates you and your interest in clinical research – specifically in rare disease?
I started my career in biology, with a special interest in plant sciences and their medicinal use in local communities in Latin America. While my enthusiasm for ethnobotany and medical plant use stayed with me, graduating from university made me realize that I wanted to apply my scientific training to human health on a larger and more applied scale.
I then moved into clinical research. Over the last decade, I have built my expertise from the ground up, moving into the clinical management of rare disease trials. In my current role, I oversee the operational complexities of these trials, which can span a multitude of different disciplines, ensuring protocols are executed rigorously while managing cross-functional teams and highly complex patient logistics.
What especially motivates me about rare diseases is that at some point, you are not just an operational manager, you become an advocate for the patients and their families. Because some of these patient populations are so small and treatment options are so limited, or even nonexistent, you get to know their struggles intimately. With every decision you make, you hope to alleviate their burden and improve their quality of life, not just on paper, but in their day-to-day realities. You are fighting to make trials accessible, manageable, and safe for families who are often exhausted from searching for answers.
What should Sponsors look for in a CRO partner when conducting a rare disease trial?
In clinical research, data integrity is everything—protocols and processes must be strictly enforced. At the same time, you need to understand the struggles of patients and their families. Accounting for their needs while ensuring 100% protocol adherence can be a massive challenge. Versatility has been crucial in the studies I have managed. In the rare and ultra-rare disease space, you can never forget that the ultimate form of patient advocacy is flawless data integrity. If the data is compromised, the treatment will not get approved, and patients lose their chance for a future treatment opportunity they desperately need.
Additionally, a collaborative, cross-functional model has been a crucial factor in the success of the rare disease clinical trials we have conducted. Based on my personal experience driving these trials to full regulatory approval, these are some key items to look for:
- A Dedicated Patient Recruitment & Retention (PRR) Team to Enhance Recruitment and Retention: In the rare disease space, finding patients is the first challenge; keeping them is the odyssey that follows. Our PRR team has been incredibly helpful in maintaining relationships with patient advocacy groups and supporting travel logistics, mitigating the trial burden on patients and their families.
- Embedded Medical Experts: Our team of Medical Monitors and Advanced Clinical Practitioners brings a wealth of knowledge from a very “hands-on” perspective. Their engagement and close oversight of the patients—while maintaining tight relationships with the Principal Investigators—have been beneficial.
- Proactive Regulatory Expertise: Navigating the intricate, ever-evolving pathways of global regulatory bodies requires a specialized map. A strong CRO partner doesn’t just react to regulatory requests; they proactively leverage special pathways, such as securing orphan drug, fast-track, or breakthrough therapy designations. They enforce rigorous compliance from day one, delivering clean, inspection-ready data packages that can successfully cross the finish line.
- Uncompromising Operational Excellence: With trials in the rare and ultra-rare space, there is little margin for error. Sponsors need a CRO capable of precision execution across complex, multi-site global trials. This means managing intricate Investigational Product (IP) logistics, temperature-controlled supply chains, and harmonizing relationships between clinical sites, vendor laboratories, and data management systems to maintain a frictionless trial ecosystem.
Ultimately, Sponsors should look for a partner who understands that in rare disease, operational rigor and human empathy aren’t competing priorities—they go hand in hand and are the foundations for success.
What has been one of your most rewarding experiences supporting a rare disease clinical trial, and why did it stand out to you?
Without a doubt, my biggest reward is seeing science successfully cross the finish line. I have been incredibly fortunate to work on trials with outstanding safety and efficacy profiles, ultimately leading to full regulatory drug approvals.
In rare disease, an approval isn’t just a corporate win; it is a historic and personal milestone. Knowing that our operational rigor, the long days, and the fierce advocacy directly contributed to putting a life-saving or life-changing drug into the hands of families who previously had zero options—that is an indescribable feeling. Seeing first-hand the stories of relief shared by the patients and their families has meant the world to me.
One particular case that touched me deeply was the story of a surgeon who was suffering from a very debilitating condition and participated in a clinical trial. This did not just stop their condition from deteriorating, it reversed the illness to a point where they could practice and perform surgery again, in turn improving other people’s lives. The drug is now available in many countries, helping a much greater number of people affected by this devastating condition. This experience encapsulates the paradigm that the ultimate form of patient advocacy is flawless clinical studies—and that at the same time, there is always room for personal empathy.
Medpace is a Leading Rare Disease CRO
Rare disease clinical trials present unique challenges in trial design, regulatory strategy, site selection, patient enrollment, and project management. Successfully navigating these complexities requires a highly experienced, full-service CRO with innovative strategies to identify and engage sites and scarce patient populations, and with expertise in site and project management to achieve study aims. Accelerate your rare disease trial with Medpace’s cross-functional team of experts, who leverage innovative study approaches and strong site relationships to support efficient trial execution.
